Title

Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex
A Phase 2/3, Multi-Center, Double-Blind, Placebo-Controlled, Randomized, Parallel-Group, Dose-Response Comparison of the Efficacy and Safety of a Topical Rapamycin Cream for the Treatment of Facial Angiofibromas (FA) Associated With Tuberous Sclerosis Complex (TSC) in Patients 6 Years of Age and Over
  • Phase

    Phase 2/Phase 3
  • Study Type

    Interventional
  • Status

    Active, not recruiting
  • Intervention/Treatment

    rapamycin ...
  • Study Participants

    107
The study aims to compare the safety and efficacy of two different strengths of Rapamycin cream, topical and placebo over 26 weeks in the treatment of facial angiofibroma (FA) associated with Tuberous Sclerosis Complex (TSC).
Topical rapamycin has previously been used to treat FA associated with TSC, reducing erythema, papule size, while flattening lesions and improving skin texture. Topical rapamycin has been reported to be well tolerated.

The efficacy and safety of a topical rapamycin cream at two strengths (0.5% and 1.0%) will be assessed during a 26 week double-blind treatment phase with assessments made at clinical visits at baseline, 2, 8, 14, 20 and 26 weeks, and at follow-up (4 weeks after the last dose of study drug).
Study Started
Jul 28
2019
Primary Completion
Apr 30
2022
Anticipated
Study Completion
May 31
2022
Anticipated
Last Update
Mar 23
2022

Drug rapamycin

Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks

  • Other names: sirolimus

Drug placebo

Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks

0.5% Rapamycin cream, topical Experimental

Rapamycin cream topical, 0.5% w/w, applied once daily before bed on affected area for 26 weeks

1.0% Rapamycin cream, topical Experimental

Rapamycin cream topical, 0.5% w/w, applied once daily before bed on affected area for 26 weeks

Placebo Placebo Comparator

Placebo cream topical, applied once daily before bed on affected area for 26 weeks

Criteria

Inclusion Criteria:

Male and female patients aged ≥ 6 years and ≤ 65 years on the day informed consent is obtained
Patients diagnosed with TSC based on the clinical diagnostic criteria of International Tuberous Sclerosis Complex Consensus Conference 2012 and presenting visible facial angiofibroma
An FA severity score of 2 or 3 on the IGA scale
Patients or their legal representatives capable of understanding the explanation of the clinical trial and who give written informed consent for participation
Patients or their legal representatives able to maintain patient diaries following the instructions of the investigator or sub-investigator

Exclusion Criteria:

Patients who cannot carry out the treatment plan or follow-up assessment
Patients with serious skin lesions such as erosions or ulcers
Patients with known hypersensitivity to any component of the study product
Patients who have received rapamycin/sirolimus, everolimus, or temsirolimus within 3 months of enrolment
Patients who received laser therapy or surgical therapy within 6 months prior to trial enrolment
Patients who participated in any other clinical trial within 3 months prior to the day of enrolment
Patients judged unsuitable for this clinical trial by the investigator or sub-investigator
Pregnant or lactating females
Sexually active females of childbearing potential not using adequate contraception and sexually active males not using adequate contraception
Patients with immune dysfunction or receiving any form of immunosuppression
Patients with severe FA, with a score of 4 on the IGA scale
Patients with an FA severity score of less than 2 on the IGA scale
No Results Posted